reflections on genomic editing in nonhuman primates (marina emborg)
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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_iblijtfhks hk `ikhnfj icftfk` fk khkaundk prfndtis
Ndrfkd I. Inohr`, N.C. ]a.C.
FLD_ 4?-=0-?=49
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Sai dkfndl nhcil dkc ahw ft fs usic fnpdjt jlfkfjdl trdksldtfhk
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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@ikhnfj Icftfk` (@I) fk khk aundk prfndtis (dkc htair spijfis)
NHCILZ SAI_D]FIZ
@I shndtfj@I `irnlfki
Dpplfjdtfhks hb @I
@I shndtfj@I `irnlfki
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Oibhri dpplyfk` @ikhnfj Icftfk` fk khkaundk prfndtis….
Pay ch wi ch risidrja fk
khkaundk prfndtis8
• Sh ukcirstdkc cfsidsi
•
Sh tist bfrst-fk-jldss dkc fkvdsfvi tairdpfis
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Nhkeiys vs. rhcikts - Oiadvfhrdl seflls
YJ Cdvfs - K]_J Nhkeiy nhcils prhvfci hutjhni nidsuris tadt advi jlfkfjdl fnpdjt
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Nhkeiys vs. rhcikts ‚ Ordfk jhnplixfty
Nhusi
_aisus Nhkeiy
Nhkeiy nhcils dllhw civilhpnikt hb fkvdsfvinitahcs dkc jdk prhvfci phst nhrtin ivfcikji hbsujjiss hr bdfluri
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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]drefkshk‖s cfsidsi (]C)
• ]C fs typfjdlly cisjrfoic ds d prh`rissfvi kiurhci`ikirdtfvi nhvinikt cfshrcir
dsshjfdtic th tai lhss hb CD kiurhks fk tai ZKpj dkc tai prisikji hb LOs dkc LKs
• Ndfkstdy hb tairdpy7 CD ripldjinikt.
_K
JJ
D`fk` ]C
ZK
Rirnflyid dkc Inohr`,, ?=49@hicirt it dl, ?==4
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Zuostdktfd Kf`rd
Jhrtix
Chrsdl Nhthr Kujlius hb tai Rd`us
Icfk`ir-Pistpadl Kujlius
]drdkf`rdl Kujlius
Ayphtadldnus
Hlbdjthry Oulo
Lhjus Jhirulius
Kujlius Odsdlfs hb Niykirt
Zirhthkir`fj _dpai Kujlif
]icukjulhphktfki Kujlius
_itfjuldr Bhrndtfhk hbtai ]hks dkc Nfcordfk
]irfpairdl Zynpdtaitfj @dk`lfd
Fktirnicfhldtirdl Jill Jhlunkhb tai Zpfkdl Jhrc
Bftzpdtrfje it dl, Dktfhx. _ic. ?==6
]C dbbijts nultfpli JKZ dkc ]KZ drids…
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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]C synpthns riblijt tai jhnplixfty hb tai cfsidsi
Zwdkshk dkc Inohr`, ?==6
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Padt jdusis ]C8
Pi ch kht ekhw wadt trf``irs]C
Dlpad sykprhtifkd``ri`dtfhk
taind`fjsahp.jhn.du
Afrsja it dl, ?=4:
Padt jdusis ]C8
_fse bdjthrs fciktfbfic7
•
D`fk`
•
Ikvfrhknikt
•
@ikitfjs
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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]usjandk, ?=4:
Duthshndl chnfkdkt
ZKJD (]D_E4, ]D_E3)
L__E? (]D_E1)
R]Z:9
IFB3@4
Duthshndl rijissfvi
]D_EFK (]D_E?)
]FKE4 (]D_E2)
CG4 (]D_E0)
@ikis rhoustly dsshjfdtic wfta ]C/]drefkshkfsn
•
Sai L__E? prhtifk fs d ldr`i (?9?0 dnfkhdjfcs) @S]-ri`uldtic sirfki/tarihkfki efkdsitadt jhktdfks sivirdl prhtifk-prhtifk fktirdjtfhk chndfks. Ft fs prisikt fk tai ordfk fk drids
eiy th ]C. Fts rhli fs kht jlidr.
• Zfkji fts cfsjhviry fk ?==3, L__E? nutdtfhks advi oiik fciktfbfic ds tai nhst jhnnhk`ikitfj jdusi hb bdnflfdl dkc khk bdnflfdl ]C (4-:=% hb dll ]C jdsis cipikcfk` hkphpuldtfhk stucfic).
• L__E?@?=46Z fs tai nhst briquikt nutdtfhk. ]dtfikts jdrryfk` tafs nutdtfhk sahw d jlfkfjdldkc kiurhpdtahlh`fjdl prhbfli fkcfstfk`ufsadoli brhn sphrdcfj ]C.
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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]C nhkeiy nhcils jurriktly dvdfldoli
NHCIL]C
itfhlh`y Kiurhprhtijtfvi _isthrdtfvi LFCKhk nhthr
sf`ks
D`fk` 4--? ? 3 3 :
Dlpad sykujlifk `ikitrdksbir 4--? 4--? 4--? 3 9
2-HACD fktrdstrfdtdl 4--? 4--? 4--? 3 9
N]S] systinfj djuti : 3 4 4 ?
N]S] systinfjfktirnicfdti ? 3 4 4 ?
N]S] systinfj jarhkfj 4--? 3 4 4 ?
N]S] fktrdjdrhtfc ? 4--? 4 3 3
N]S] fktrdjdrhtfc + fv : : 4 ? :
Nhcil rdtfk`7 4- nhst suftdoli, 9-fkdciqudti
Jdrcfdj cysduthkhnfd nhcil fk ]C oy systinfj 2-HACD
Inohr`, FLD_ ?==0
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Lfnftdtfhks hb jurrikt nhcils
Dkfndl nhcils ]C
Kiurhjainfjdl bhjus CD CD++, out dlsh KD, 9AS, Dja
Kiurhdkdthnfjdl bhjus ZKj ZKj, ZKr dkc htair JKZ dkc ]KZ systins
Jilluldr pdtahlh`y jill lhss jill lhss, liwy ohcfis
]dtah`ikisfs thxfj ukekhwk thxfj x `ikitfj fktirdjtfhk
Sfni jhursi djuti slhw prh`rissfvi
_ijhviry sf`kfbfjdkt (ukliss lisfhk jhnpliti) prh`rissfvi hkji jhnpiksdtfhk ixadustic
Htair
OOO hpikfk` dsshjfdtic th tridtnikt,`lfdl sjdrs fk fkgijtfhk sftis ****
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Liktfvfrdl-odsic7
•
@B] raisus (Jadk it dl., ?==45 Phlb`dk`it dl., ?==45 Kfu it dl, ?=4=) ndrnhsit(Zdsdef it dl., ?==6).
•
Auktfk`thk‖s cfsidsi @B]+JD@ raisus
auktfk`tfk ripidt (Vdk` it dl, ?==1)
•
]C aundk D9:S-syk jykhnhl`hus (Kfu it
dl., ?=49).
Jhnpdric th htair spijfis KA] trdks`ikisfs ads ld``ic
J_FZ]_-odsic7•
]pdr-` dkc _d`4 (Kfu it dl, ?=43)
• Cujaikki sykcrhni (Jaik it dl, ?=49)• Q-lfkeic dcrikdl ayphpldsfd jhk`ikftd
(DAJ) dkc ayph`hkdchtrhpfj
ayph`hkdcfsn (AA), Cdx4 ekhjehut
jykhnhl`hus bitus (Edk` it dl, ?=42)
…prh`riss,
Ndppfk` aundk dkc khk aundk `ikhni,
Fciktfbyfk` cfsidsi-dsshjfdtic ̀ ikis,
Lfnftdtfhks hb jurrikt nhcils dkc tairdpfis, dri builfk` jadk`i…
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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]usjandk, ?=4:
Duthshndl chnfkdkt
ZKJD (]D_E4, ]D_E3)
L__E? (]D_E1)
R]Z:9
IFB3@4
Duthshndl rijissfvi
]D_EFK (]D_E?)
]FKE4 (]D_E2)
CG4 (]D_E0)
@ikis rhoustly dsshjfdtic wfta ]C/]drefkshkfsn
• Pfta ixjiptfhk hb ZKJD trfshny, ]C ads oiik lfkeic th sfk`li phfkt nutdtfhks.
•
Safs su``ists tadt tai jldssfj dpprhdja hb trdks`ikfj hvirixprissfhk hb d nutdticprhtifk ndy oi liss ibbijtfvi dt prhcujfk` tai aundk ]C paikhtypi tadk fktrhcujfk` tai
fciktfjdl dllilfj vdrfdkt bhukc fk tai aundk cfsidsi-dsshjfdtic dllili.
• @I jdk prhvfci d shlutfhk.
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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@ikhnfj Icftfk` (@I) fk khk aundk prfndtis (dkc htair spijfis)
NHCILZ SAI_D]FIZ
@I shndtfj@I `irnlfki
Dpplfjdtfhks hb @I
@I shndtfj@I `irnlfki
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
17/31
]usjandk, ?=4:
Duthshndl chnfkdkt
ZKJD (]D_E4, ]D_E3)
L__E? (]D_E1)
R]Z:9
IFB3@4
Duthshndl rijissfvi
]D_EFK (]D_E?)
]FKE4 (]D_E2)
CG4 (]D_E0)
Padt `iki fs rilivdkt th tai cfsidsi8
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Ndrnhsit dt cdy 49Zjaultz-Cdreik it dl, ?=49
_aisus dt cdy 49
Padt spijfis8
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Inoryh
Srdksbir
rijfpfikt
FRB/FJZF
L__E? @?=46Z
Srdks`ikfj Ndrnhsits bhr Srdksldtfhkdl Ztin Jill _isidrja
f]ZJ
Sairdpiutfj
jills
D J_FZ]_ dpprhdja
Wy`htfj jythpldsnfj fkgijtfhk
@ikhnfj icftichbbsprfk`
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Zjaultz-Cdreik it dl., Civ ]syja. ?=49
Jhnnhk ndrnhsit nhkeiys civilhpniktdl nflisthkis
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Ch khknhthr sf`ks pricfjt ]C8
Khripfkiparfki lhss & fkjridsi fk thxfj nitdohlftis
ricujic RNDS? ixprissfhk
Lhjus jhirulius
Kihjhrtix
JKZ7 fnpdfric jh`kftfvi bukjtfhks, ciprissfhk,sliip cfsturodkji, dkxfity
Jdrcfdj/duthkhnfj7 hrtahstdtfj ayphtiksfhk, bdtf`ui, cysraytanfds
Fkftfdti padrndjhlh`fjdl hr lfbistyli strdti`fis th dttikudti burtair chpdnfkilhss & ricuji thxfj nitdohlftis
"#$#%$&'( ') %'*(&$&+# ,&*(, - '$.#/ =&,'/=#/, 4$ 4*# /4(*#
') */#4$#,$ ?" /&,:3 924*&(* ') &204&/#= #1#%6$&+#
)6(%$&'(3 "#$#%$&'( ') $'1&% 2#$47'5&$# &( 0#/&0.#/
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@ikhnfj Icftfk` (@I) fk khk aundk prfndtis (dkc htair spijfis)
NHCILZ SAI_D]FIZ
@I shndtfj@I `irnlfki
Dpplfjdtfhks hb @I
@I shndtfj@I `irnlfki
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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@B] JC21 JC: JC1
@BD]
(kh kfssli)
K f ` r d
] u t d n i k
ALD-C_
(kh kfssli)(kh Kfssl jhuktirstdfkfk`)
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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8/20/2019 Reflections on Genomic Editing in Nonhuman Primates (Marina Emborg)
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Jill ripldjinikt fs nhri tadk pldyfk` d puzzli…
Ofk` fnd`is
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Ahst dkc chkhr advi d rhli fk `rdbt ibbfjdjy
Bftzpdtrfje it dl, Dktfhx. _ic. ?==6
• Jill ripldjinikt fk d @I ]C nhcil prhvfci dk hpphrtukfty th dssiss tai fnpdjt hb
nutdtfhks fk jill survfvdl
• @I Jill `rdbts jdk dlsh dllhw th dssiss dltirkdtfvis bhr pirshkdlfzic nicfjfki
• @I bhr C_IDCC dpprhdja th nhculdti F]ZJs-cirfvic chpdnfki kiurhks djtfvfty
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@ikhnfj Icftfk` (@I) fk khk aundk prfndtis (dkc htair spijfis)
NHCILZ SAI_D]FIZ
@I shndtfj@I `irnlfki
Dpplfjdtfhks hb @I
@I shndtfj@I `irnlfki
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….hb aundks dkc khk aundk prfndtis
attp7//www.ouflcdoidr.jhn
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Jhksfcirdtfhks OIBH_I dpplyfk` @I bhr KA] cfsidsi nhcilfk`
•
Cfsidsi-rildtic fssuis7 nutdtfhk,
d`i hb hksit, synpthns
•
Zpijfis7 `ikhni, lfbispdk,
civilhpniktdl pdttirks,
ahusfk` kiics, oriicfk` cindkcs,
fnpdjt hb fkcfvfcudl `ikitfj
odje`rhukc (hutoric vs. fkoric)
•
Sistfk`7 spijfbfj th cfsidsi, d`i,
spijfis
•
@I-rildtic fssuis7 nitahcs bhr pldsnfc
fkhjuldtfhk, ukfplix vs. nultfplix,rdkchn fksirtfhks/cilitfhks, hbb
tdr`it ibbijts, nhsdfjfsn Rirnflyid dkc Inohr`, G Kiurhsjf Nita ?=49
Eiip fk nfkc “wadt fb„ sjikdrfh dkc oi ridcy wfta jlfkfjdl
prfndti vitirfkdrfdk supphrt.
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]rdjtfjis tadt ailp istdolfsa itafjdl odsfs bhr fkftfdtfk` bfrst-fk-aundk trfdls
Efnnilndk it dl, Nhv Cfs. ?==6
Jhkjirk ]rdjtfjis
Fktirkdl vdlfcfty D prfhrf ayphtaisfs stdtic
D prfhrf phwir jdljuldtfhks
_dkchnfzdtfhk
Olfkcic tridtnikt dllhjdtfhk
Olfkcic hutjhni dssissnikts
Cfsjussfhk hb nfssfk` cdtd
Ixtirkdl vdlfcfty Ixpldkdtfhk bhr silijtfhk hb dkfndl nhcil
Ixpldkdtfhk hb silijtfhk hb hutjhni nidsuris
Jhrrisphkcikji hb trfdl wfta prijlfkfjdljhkcftfhks
Hptfnfsn dkc Jrftfjdl rivfiw hb prijlfkfjdl cdtd
puolfjdtfhk ofds Bull puolfjdtfhk hb prijlfkfjdl stucfis
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Zpijfdl Sadkes
Safs risidrja wds supphrtic oy `rdkts brhn tai YP FJS_,YP RJ_@I dkc tai KFA
Tuistfhks8 inohr`Mprfndti wfsj icu
YP - Ndcfshk7
Zjhtt Rirnflyid
Zjhtt @utarfiEdt Orduk
Sic @hlhs
Gikkd Erhpp
@ri` Pfipz
Nfjadil Niyir
Zu-Jauk Wadk`
Gdnfi Sahnshk
Kdkjy Zjaultz-Cdreik
Ghk Livfki
“Ouccy„ Jdpudkh
KFA7
Ndre Jhheshk
Jdrki``fi Nillhk
Dlix Lhkchk
Ndj@fll Ykfvirsfty7
Ghkdtadk Efnnilndk
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